49P Epidemiologic study of XL-MTM and clinical expression in the liver (EXCEL): study design of an observational, patient-centric study.

Autor: Beggs, A.¹ (AUTHOR), Haselkorn, T.² (AUTHOR), Dhawan, A.³ (AUTHOR), Lawlor, M.⁴ (AUTHOR), Kim, J.² (AUTHOR), Ward, E.⁵ (AUTHOR), Hughes, Z.⁶ (AUTHOR), Baima, J.² (AUTHOR), James, L.² (AUTHOR), Coats, J.² (AUTHOR), Gentyala, R.² (AUTHOR), Brandon, T.² (AUTHOR), Garcia-Malo, M. Jesus Perez Inigo⁷ (AUTHOR), Marini Bettolo, C.⁸ (AUTHOR), Graham, R.⁹ (AUTHOR), Dowling, J.¹⁰ (AUTHOR)

Publikováno v: Neuromuscular Disorders. 2024 Supplement 1, Vol. 43, pN.PAG-N.PAG. 1p.